Rationale: Polycystic liver disease (PLD) is a rare disorder characterized by >20 fluid-filled hepatic cysts. Polycystic livers are present in the combination with renal cysts as a manifestation of autosomal dominant polycystic kidney disease (ADPKD), or isolated in the absence of renal cysts as autosomal dominant polycystic liver disease (ADPLD or PCLD). PLD patients are confronted with symptoms caused by the mass effect of their polycystic liver every day for the rest of their life. There is no standard therapeutic option for symptomatic PLD patients. Current options are fairly invasive or their efficacy is only moderate.

Preliminary data in our research lab have shown that ursodeoxycholic acid (UDCA) inhibited the proliferation of polycystic human cholangiocytes in vitro through the normalization of the intracellular calcium levels in cystic cholangiocytes. The investigators also found that daily oral administration of UDCA for 5 months to PCK rats, an animal model of ARPKD that spontaneously develops hepato-renal cystogenesis, resulted in inhibition of hepatic cystogenesis.

The investigators hypothesize that UDCA is an effective therapeutic tool in reducing liver volume in PLD.

Objective: First, to demonstrate whether UDCA-therapy is effective in reducing total liver volume in PLD patients. Second, the investigators want to assess if UDCA modifies quality of life. Finally, the investigators want to assess safety and tolerability.

Symptomatic defined as ECOG-PS ≥ 1 (2), and having at least three out of ten PCLD symptoms:

Informed consent, patients are willing and able to comply with the study drug regimen and all other study requirements.

Exclusion Criteria:

Use of oral anticonceptives or estrogen supplementation

Use of UDCA in 3 months before baseline

Females who are pregnant or breast-feeding or patients of reproductive potential not employing an effective method of birth control.

Intervention (aspiration or surgical intervention) within six months before baseline

Treatment with somatostatin analogues within six months before baseline

Renal dysfunction (MDRD-GFR < 30 ml/min/1.73m2)

Patients with a kidney transplant

Hypersensitivity reaction to UDCA or patients with galactose-intolerance, lactase deficiency or glucose-galactose malabsorption

Acute cholecystitis or frequent biliary colic attacks

Acute stomach or duodenal ulcers

Inflammation of small intestine or colon

Use of drugs that can interact with UDCA, such as colestyramine, aluminium hydroxide or cyclosporin

Enrolment in another clinical trial of an investigational agent while participating in this study

History or other evidence of severe illness or any other conditions which would make the patient, in the opinion of the investigator, unsuitable for the study

Mental illness that interferes with the patient ability to comply with the protocol

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Please refer to this study by its ClinicalTrials.gov identifier: NCT02021110